The technology potentially has applications in the development of new cell therapies and will augment Horizon’s research tools and services.
Horizon will collaborate with Rutgers University to further develop the novel base editing platform from the laboratory of Dr. Shengkan Jin, associate professor of pharmacology at Rutgers Robert Wood Johnson Medical School. As part of the agreement, Horizon has made a non-material payment to Rutgers for an option to exclusively license the base editing technology for use in all therapeutic applications. As part of the collaboration, Horizon will also fund further research in base editing at Rutgers University while undertaking evaluation and proof of concept studies at Horizon.
Base editing is a novel technology platform for engineering DNA or genes in cells that has the potential to correct errors or mutations in the DNA by modifying genes using an enzyme. Compared with currently available gene editing methodologies such as CRISPR/Cas9, which creates “cuts” in the gene that can lead to adverse or negative effects, this new technology allows for more accurate gene editing while reducing unintended genomic changes. The technology will have a significant impact in enabling cell therapies to be progressed through clinical development and towards commercialisation. For more scientific detail on base editing, please see below in the notes to editors.
Terry Pizzie, Horizon’s Chief Executive Officer, said: “Base editing is potentially transformative for all gene editing technologies with the potential to help target many diseases that to date have no treatment. As a world leader in the field of gene editing and gene modulation, both in research and applied markets, we are very excited to partner with Dr Jin and Rutgers University. By extending our scientific and IP capabilities, Horizon will now be able to more fully support our pharma, biotech and academic partners to deliver better cell therapy solutions to patients. As part of our five-year investment strategy, Horizon committed to investing in high value technologies that maintain our market leadership; base editing technology is a perfect example of that. We look forward to updating on the progress of the partnership and potential future next steps.”
Dr. Shengkan ‘Victor’ Jin of Rutgers University stated: “The cytidine deaminase version of the technology alone could potentially be used for developing ex vivo therapeutics such as gene modified cells for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, and over-the-shelf CAR-T cells for leukemia, as well as in vivo therapeutics for inherited genetic diseases. The potential is enormous. In addition to the ‘simple’ diseases caused by a single genetic alteration event, the therapeutic strategy, in principle, could also be useful for treating diseases where permanently targeting a disease-related gene is beneficial.”
Dr. David Kimball, Interim Senior Vice President for Research and Economic Development at Rutgers University, added: “Gene editing technology has truly revolutionised how scientists think about their search for better options and outcomes in the treatment of disease. We look forward to advancing the shared goals of further developing this novel base editing platform and improving human health through this collaboration with Horizon.”